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Regeneron halts Phase 1 study into ultra-rare disease due to patient deaths

Published on 03/11/20 at 01:20pm

Regeneron has slammed the brakes on its Phase 1 trial of garetosmab in the treatment of the ultra-rare condition fibrodysplasia ossificans progressiva (FOP) due to fatal serious adverse events reported in the open-label extension of the trial where all participants were administered with active treatment.

In a review of 28-week findings reported in January evaluating responses in 44 patients between the ages of 18 and 60, garetosmab was shown to decrease total lesion activity by 25% and flare-ups by half over placebo. No deaths were reported during the 28-week double-blind treatment period.

“Accumulation of heterotopic ossification (HO) across the body is the defining characteristic of FOP and severely limits physical function over time,” remarked Dr Robert Pignolo, Division of Geriatric Medicine and Gerontology, Department of Internal Medicine at the Mayo Clinic. “The MOVE trial provides important insight into long-awaited treatment strategies and demonstrates that the oral therapy palovarotene can reduce new HO volume, representing an important therapeutic option in FOP especially in older children and adults.”

The findings have been reported to an Independent Data Monitoring Committee and global regulators, with a review of the trial due to be conducted.

Affecting around 1.3 million people across the world, FOP is characterised by soft tissue flare-ups that can lead to HO, whereby muscle, tendon and ligament tissue is progressively converted into bone tissue, severely debilitating patients.  

“This disease is relentless and devastating, leaving many patients wheelchair-bound or locked in a position unable to move, with a dramatically curtailed lifespan,” commented Dr George D Yancopoulos, President and Chief Scientific Officer at Regeneron. “We believe garetosmab may offer important new hope that can potentially transform the course of FOP and look forward to working closely with the FDA and other regulatory authorities to make garetosmab available.”

Matt Fellows

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